Q: What prompted the creation of the foundation?
My wife Marnie and I decided to form the Adenoid Cystic Carcinoma Research Foundation (ACCRF) in the aftermath of her diagnosis and treatment in 2004. We learned that adenoid cystic carcinoma (ACC) is a rare cancer of the secretory glands, usually found in the head and neck region. And we came to know that the course of ACC can be slow yet relentless, with many patients experiencing recurrences several years after surgery and radiation having eradicated any evident disease.
We wanted to do something to help improve treatments and, ideally, find a cure. But, when we searched for an organization to support that was focused primarily on ACC research, we couldn't find one. There were only sporadic and uncoordinated projects from a few researchers who spent a small percentage of their time on ACC. We felt we could help change this situation for the better.
ACCRF has three categories of projects to address our three broad challenges. First, as with most rare diseases, ACC suffered from a paucity of specimens and models that kept researchers from working their magic. Second, the molecular understanding of ACC biology was minimal, so there was nothing to target in a rational manner. And, lastly, there was no plan for translating what was known about ACC biology into novel therapeutic approaches and reasonable concepts for clinical research.
Q: How has the foundation tried to overcome those barriers?
We drafted a strategy focused on creating a community of interested researchers who follow a coordinated plan of action with the active support of the patient community. Our research agenda has been central to galvanizing the research effort. Rather than passively review investigator-initiated proposals, ACCRF's Scientific Advisory Board laid out a program of directed research to put in place the essential building blocks for success. We seek out investigators with the expertise, interest, and capacity to address research questions that we know must be answered. We don't wait for promising ideas to float in over the transom. And, clear guiding principles govern how we interact with stakeholders and build our research portfolio.
Both patients and researchers have responded well to this approach. Patients have responded by donating specimens and financial resources, as well as accruing to clinical trials with clear rationales. Researchers have been energized to know that their promising findings will be carried forward towards the clinic.
We'd hesitate to label accomplishments as belonging only to ACCRF, as so many researchers and patients have walked the journey by our side. The most gratifying achievements of our ACC research community since the foundation's inception nearly seven years ago are:
- Development of biorepository resources with hundreds of high-quality specimens
- Discovery of ACC cell line misidentifications
- Creation of substantial xenograft resources
- Generation of genomic datasets across multiple platforms from the same specimens
- Discovery of a highly recurrent translocation that drives most ACCs
- Preclinical drug screening of approved and novel agents in xenografts
- Coordination with the National Institute of Dental and Craniofacial Research to increase NIH focus on and funding for salivary gland cancer research
- Initiation of the first-ever clinical trial in ACC with a clear scientific rationale and supportive preclinical drug screening data
Our continuing top research goal is to generate a portfolio of information that will lead to more clinical trials with high likelihoods of success. I suppose you could say we seek to provide our partners, whether private industry or the NIH, with "complete translational stories" on a silver platter, making it easy for them to allocate capital and accrue patients.
- High-throughput screening of zebrafish to identify compounds that suppress the driver of ACC
- Identification of druggable targets downstream of the driver of ACC
- Functional studies of pathways newly-identified as significant in ACC by exome sequencing
- Further drug screening in xenografts models with inhibitors of potential targets
- Validation of a transgenic model of ACC
We really have been fortunate to work with such a great group, from the patients and donors to the researchers and clinicians. And, with the continued commitment and coordination of our growing ACC research community, we are confident in the eventual success of our efforts.